Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy

NCT06018558 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: OCU410-101
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 12

Brief Summary

This is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration (AMD). This is a multicenter study, which will be conducted in two phases and will enroll up to a total of 60 subjects.

Conditions

Geographic Atrophy

Outcome Measures

Primary Outcomes (6)

• Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events))

  The primary endpoint is safety, determined by the number of ocular and non-ocular Study Drug-related adverse events (SDAE), treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs).

  12 months (Screening to 12 months post OCU410 administration)

• Change in anatomy of ocular structures using Slit Lamp Biomicroscopy

  We will use Slit-lamp Biomicroscopy to visualize the anatomy of ocular structures before and after sub-retinal injections and follow-up visits.

  12 months (Screening to 12 months post OCU410 administration)

• Change in anatomy of ocular structures using Indirect ophthalmoscopy

  We will use Indirect ophthalmoscopy to visualize the anatomy of ocular structures before and after sub-retinal injections and follow-up visits.

  12 months (Screening to 12 months post OCU410 administration)

• Change from baseline in BCVA (Best Corrected Visual Acuity)

  Visual function of the study eye was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) Best Corrected Visual Acuity (BCVA) letter score. A higher score represents better vision.

  12 months (Screening to 12 months post OCU410 administration)

• Change in Low Luminance Visual Acuity

  Measured by letter score. A higher score represents better vision

  12 months (Screening to 12 months post OCU410 administration)

• Change in the Intraocular Pressure (mmHg)

  Measured by applanation or rebound tonometry with confirmation with Goldmann tonometer if IOP is outside normal range (8-21mmHg).

  12 months (Screening to 12 months post OCU410 administration)

Secondary Outcomes (3)

• Humoral and cellular immune response

  12 months (Screening to 12 months post OCU410 administration)

• Shedding of viral vector

  12 months (Screening to 12 months post OCU410 administration)

• Laboratory parameters including serum chemistry and hematology

  12 months (Screening to 12 months post OCU410 administration)

Other Outcomes (5)

• Structural Outcome: Change Using Qualitative and quantitative assessments of autofluorescence pattern (FAF)

  12 months (Screening to 12 months post OCU410 administration)

• Changes in National Eye Institute Visual Function Questionnaire 25 (NEI-VFQ25)

  12 months (Screening to 12 months post OCU410 administration)

• Change From Baseline in Mean Threshold Sensitivity (MAIA)

  12 months (Screening to 12 months post OCU410 administration)

Study Arms (6)

Phase1 Dose Escalation- Low Dose (2.5×10E10 vg/mL):

EXPERIMENTAL

Low Dose (2.5×10E10 vg/mL): Subjects will receive a subretinal injection of OCU410 in the low dose concentration.

Genetic: OCU410

Phase1 Dose Escalation- Medium Dose (5×10E10 vg/mL):

EXPERIMENTAL

Medium Dose (5×10E10 vg/mL): Subjects will receive a subretinal injection of OCU410 in the medium dose concentration.

Genetic: OCU410

Phase1 Dose Escalation- High Dose (1.5×10E11 vg/mL):

EXPERIMENTAL

High Dose (1.5×10E11 vg/mL): Subjects will receive a subretinal injection in the high dose concentration.

Genetic: OCU410

Phase 2 Dose Expansion: Maximum tolerated dose (MTD) from Phase 1-Randomized Arm

EXPERIMENTAL

Maximum tolerated dose (MTD) from Phase 1: Subjects will receive a subretinal injection in the MTD concentration.

Genetic: OCU410

Phase 2 Dose Expansion: Lower Dose from Phase 1-Randomized Arm

EXPERIMENTAL

Subjects will receive a subretinal injection of OCU410 in a Lower Dose concentration.

Genetic: OCU410

Control Arm

NO INTERVENTION

No Intervention Control Arm: Subject will not receive any active study intervention

Interventions

OCU410 GENETIC

Subretinal administration of OCU410

Phase 2 Dose Expansion: Lower Dose from Phase 1-Randomized Arm; Phase 2 Dose Expansion: Maximum tolerated dose (MTD) from Phase 1-Randomized Arm; Phase1 Dose Escalation- High Dose (1.5×10E11 vg/mL):; Phase1 Dose Escalation- Low Dose (2.5×10E10 vg/mL):; Phase1 Dose Escalation- Medium Dose (5×10E10 vg/mL):

Eligibility Criteria

• Age: 50 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects 50 years of age or older.
• BCVA of approximately 21 letters or more using Early Treatment Diabetic Retinopathy Study (ETDRS) chart (20/320 Snellen equivalent).
• Fundus autofluorescence (FAF) imaging shows:
• Total GA area ≥2.0 and ≤20.5 mm2 (1 and 8 disk areas \[DA\], respectively)
• If GA is multifocal, at least one focal lesion must be ≥1.25 mm2 (0.5 DA), with the overall aggregate area of GA as specified above in 3.a
• The entire GA lesion must be completely visualized on the macula-centered image and must be able to be imaged in its entirety, and not contiguous with any areas of peripapillary atrophy
• Presence of any pattern of hyper-autofluorescence in the junctional zone of GA
• Subjects who had prior treatment with an approved drug for AMD, e.g. Izerway® (Avacincaptad pegol) or Syfovre® (Pegcetacoplan injection) can be included, after a washout period of at least 3 months in study eye. Subjects can receive an approved drug for AMD in the fellow eye, if required.

You may not qualify if:

• Previous treatment with a gene-therapy or cell therapy product
• Spherical equivalent of the refractive error demonstrating \> 6 diopters of myopia or an axial length \>26 mm, inability to fixate, uncontrolled glaucoma, advanced cataract, corneal abnormalities, medium haze, and other retinal pathologies.
• Any history or current evidence of exudative ("wet") AMD including any evidence of retinal pigment epithelium rips, branch retinal artery or vein occlusion, corneal transplant, or evidence of neovascularization anywhere in the retina based on fluorescein angiogram.

Sponsors & Collaborators

• Ocugen: lead

Study Sites (12)

Associated Retina Consultants

Phoenix, Arizona, 85020, United States

Location

Advanced Research, LLC

Coral Springs, Florida, 33067, United States

Location

Miidwest Eye Institute

Carmel, Indiana, 46290, United States

Location

Mississippi Retina Associates

Jackson, Mississippi, 39202, United States

Location

The Retina Institute

St Louis, Missouri, 63128, United States

Location

Mid Atlantic Retina

Cherry Hill, New Jersey, 08034, United States

Location

Duke Eye Center

Durham, North Carolina, 27710, United States

Location

The University of Pittsburgh

Pittsburgh, Pennsylvania, 15260, United States

Location

B) Retina Consultants of Texas

Bellaire, Texas, 77401, United States

Location

A) Retina Foundation of the Southwest

Dallas, Texas, 75231, United States

Location

Valley retina Institute

McAllen, Texas, 78503, United States

Location

Gundersen Health System

La Crosse, Wisconsin, 54601, United States

Location

MeSH Terms

Conditions

Geographic Atrophy

Condition Hierarchy (Ancestors)

Macular Degeneration; Retinal Degeneration; Retinal Diseases; Eye Diseases

Study Officials

• Murthy Chavali, Ph.D

  Ocugen

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Masking Details: The following team members will be masked: Bio-Statistician, Data Programmer, Imaging Reading Center Team, Head of Clinical Development and Medical Affairs.
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Study will be conducted in 2 phases: Phase 1 will be 3+3 design will be used for sequential dose-escalation cohorts in which subjects will receive a single subretinal injection of OCU410 in the study eye. Phase 2 is a dose-expansion phase of the study, where up to 51 subjects will be randomized in 1:1:1 ratio to either two OCU410 dose groups (n=17 per group) or to an untreated control group (n=17).

Study Record Dates

• First Submitted: June 30, 2023
• First Posted: August 30, 2023
• Study Start: February 23, 2023
• Primary Completion: February 17, 2026
• Study Completion (Estimated): May 29, 2026
• Last Updated: December 5, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (12)